On the Quest to Transform Cancer Care: Developments in Cell & Gene Therapies
Dec 03, 2015
By Usman (Oz) Azam, Global Head of Cell & Gene Therapies Unit (CGTU), Novartis
Over the past several years, science has provided mounting evidence supporting cell and gene therapies as the next transformative pillar of medicine. We’ve seen major advances in the field, some of which could significantly change the way we treat certain deadly cancers.
At Novartis, we’re collaborating with the University of Pennsylvania (Penn) to explore the potential of chimeric antigen receptor T cell (CART) therapies, or CARTs, to harness the power of a patient’s immune system with the aim of fighting cancer. Unlike small molecules or even other biologics, CARTs are specifically created for each individual patient through a process that reprograms their own T cells, which are designed to “hunt” and potentially kill B cells, some of which may cause cancer. It is groundbreaking science with the potential to make a meaningful difference for critically ill patients who have run out of other options.
At this year’s ASH Annual Meeting, investigators from Penn and the Children’s Hospital of Philadelphia will present exciting new data on the safety and efficacy of CTL019 in patients with different relapsed/refractory (r/r) blood cancers. In addition to longer-term data in children and young adults with r/r acute lymphoblastic leukemia (ALL), they will share findings in adult patients with certain types of r/r non-Hodgkin lymphomas: diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL) and follicular lymphoma (FL).
I am pleased to share that Novartis has opened its own global trials in r/r ALL and DLBCL with CTL019 that is being manufactured at our state-of-the-art cell processing facility in Morris Plains, NJ. It is the first FDA-approved Good Manufacturing Practice (GMP) facility of its kind in the US. At ASH, our Head of Manufacturing Jeff Boyd will present a poster that explains how Novartis took a process designed for a small academic lab and scaled it up to service global clinical trials. The latest news from Novartis in this space is that we have expanded our clinical trial footprint beyond the US and are now able to offer access to CTL019 clinical trials in Europe, Australia and Canada.
CART therapy shows a lot of promise, but there is still a lot to learn about appropriate disease targets, safety measures and ways of harnessing T-cells to target disease. Additional presentations at ASH will expand our growing understanding of CARTs, including data on CTL019 in patients who have cancer within the central nervous system; a poster exploring management of a known side effect, cytokine release syndrome (CRS); and oral presentations addressing the potential of two other CARTs – humanized CART and CART 123.
We are in uncharted territory and the future of CARTs and cell and gene therapies is still unfolding, but as the body of data grows, so does our conviction that we are on the cusp of something that may be transformative that has the potential to help patients around the world.